CLINICAL TRIALS PROFILE FOR ILOPROST

All Clinical Trials for ILOPROST

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00004786 ↗	Phase III Randomized, Double-Blind, Placebo-Controlled Study of Oral Iloprost for Raynaud's Phenomenon Secondary to Systemic Sclerosis	Completed	University of Pittsburgh	Phase 3	1995-12-01	OBJECTIVES: I. Evaluate the safety and efficacy of oral iloprost, a prostacyclin analog, in patients with Raynaud's phenomenon secondary to systemic sclerosis.
NCT00004786 ↗	Phase III Randomized, Double-Blind, Placebo-Controlled Study of Oral Iloprost for Raynaud's Phenomenon Secondary to Systemic Sclerosis	Completed	National Center for Research Resources (NCRR)	Phase 3	1995-12-01	OBJECTIVES: I. Evaluate the safety and efficacy of oral iloprost, a prostacyclin analog, in patients with Raynaud's phenomenon secondary to systemic sclerosis.
NCT00084409 ↗	Iloprost in Preventing Lung Cancer in Patients at High Risk for This Disease	Completed	National Cancer Institute (NCI)	Phase 2	2001-11-01	RATIONALE: Chemoprevention therapy is the use of certain drugs to try to prevent the development or recurrence of cancer. Iloprost may be effective in preventing lung cancer. PURPOSE: This randomized phase II trial is studying how well iloprost works in preventing lung cancer in patients who are at high risk for this disease.
NCT00084409 ↗	Iloprost in Preventing Lung Cancer in Patients at High Risk for This Disease	Completed	University of Colorado, Denver	Phase 2	2001-11-01	RATIONALE: Chemoprevention therapy is the use of certain drugs to try to prevent the development or recurrence of cancer. Iloprost may be effective in preventing lung cancer. PURPOSE: This randomized phase II trial is studying how well iloprost works in preventing lung cancer in patients who are at high risk for this disease.
NCT00086463 ↗	Trial of Iloprost Inhaled Solution as Add-On Therapy With Bosentan in Subjects With Pulmonary Arterial Hypertension (PAH)	Completed	Actelion	Phase 2	2004-06-01	The purpose of this study is to determine the safety and efficacy of Iloprost in subjects that have Pulmonary Arterial Hypertension who are concurrently taking bosentan (Tracleer TM).
NCT00109681 ↗	Inhaled Iloprost in Adults With Abnormal Pulmonary Pressure and Associated With Idiopathic Pulmonary Fibrosis	Completed	Actelion	Phase 2	2005-04-01	The primary purpose of this study is to determine whether iloprost inhalation solution is safe in subjects with idiopathic pulmonary fibrosis (IPF) and elevated pulmonary arterial pressure. The secondary purpose is to evaluate the effectiveness of this treatment in subjects with this disease.
NCT00120380 ↗	Combination Therapy of Bosentan and Aerosolized Iloprost in Idiopathic Pulmonary Arterial Hypertension (IPAH)	Terminated	Hannover Medical School	Phase 4	2004-09-01	The purpose of this study is to test whether the addition of inhaled iloprost is safe and effective in patients with idiopathic pulmonary arterial hypertension who are already being treated with the endothelin receptor antagonist bosentan.

Clinical Trial Conditions for ILOPROST

Condition Name

Condition Name for ILOPROST
Intervention	Trials
Pulmonary Hypertension	23
Pulmonary Arterial Hypertension	22
Hypertension, Pulmonary	8
Chronic Obstructive Pulmonary Disease	6

Condition MeSH

Condition MeSH for ILOPROST
Intervention	Trials
Hypertension	54
Hypertension, Pulmonary	38
Pulmonary Arterial Hypertension	31
Familial Primary Pulmonary Hypertension	28

Clinical Trial Locations for ILOPROST

Trials by Country

Trials by Country for ILOPROST
Location	Trials
United States	217
Germany	43
China	14
Japan	11
Denmark	8

Trials by US State

Trials by US State for ILOPROST
Location	Trials
California	16
Pennsylvania	13
Texas	12
New York	12
Illinois	11

Clinical Trial Progress for ILOPROST

Clinical Trial Phase

Clinical Trial Phase for ILOPROST
Clinical Trial Phase	Trials
PHASE2	1
PHASE1	2
Phase 4	13

Clinical Trial Status

Clinical Trial Status for ILOPROST
Clinical Trial Phase	Trials
Completed	54
Terminated	14
Unknown status	11

Clinical Trial Sponsors for ILOPROST

Sponsor Name

Sponsor Name for ILOPROST
Sponsor	Trials
Actelion	17
Bayer	16
Yonsei University	5

Sponsor Type

Sponsor Type for ILOPROST
Sponsor	Trials
Other	92
Industry	47
NIH	4

Clinical Trials Update, Market Analysis, and Projection for Iloprost (2023)

Last updated: January 27, 2026

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Summary

Iloprost, a synthetic analog of prostacyclin (PGI2), is approved primarily for pulmonary arterial hypertension (PAH) and the treatment of secondary Raynaud’s phenomenon. Over the past year, ongoing clinical trials have sought to broaden its indications, including potential use in connective tissue diseases and chronic thromboembolic pulmonary hypertension (CTEPH). Market dynamics reflect increasing adoption driven by expanding indications, growing prevalence of PAH, and novel formulations. This report synthesizes recent clinical trial activities, evaluates current market trends, and projects future developments up to 2030.

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Clinical Trials Update for Iloprost

Current and Recent Clinical Trials Overview (2022-2023)

Trial ID	Phase	Condition	Objectives	Status	Estimated Completion	Sponsor	Location
NCT05148263	Phase II	Scleroderma-Associated PAH	Evaluate efficacy & safety of inhaled iloprost	Recruiting	2024 Q2	United Therapeutics	USA
NCT04656314	Phase III	Chronic Thromboembolic Pulmonary Hypertension (CTEPH)	Assess inhaled iloprost as adjunct	Active but not recruiting	2024 Q4	Bayer	Germany
NCT04917267	Early Phase	COVID-19-related Pulmonary Complications	Investigate vascular effects of iloprost	Completed	2022 Q4	Academic	Multiple

Key Clinical Trial Milestones

• ILOPROST Efficacy in Connective Tissue Disease-Associated PAH (NCT05148263): Aimed at expanding IL approval scope beyond idiopathic PAH.
• Combination Therapy Trials (NCT04656314): Focus on integrating inhaled iloprost with other pulmonary hypertension agents, assessing improvements in exercise capacity and pulmonary hemodynamics.
• Safety/Tolerability in Chronic Use: Recent studies promoting dosing flexibility and inhalation device improvements.

Regulatory and Market Impact

While iloprost is well-established in PAH, recent clinical trials aim to demonstrate benefits in:

• Connective Tissue Disease-Related PAH
• Chronic Thromboembolic Pulmonary Hypertension (CTEPH)
• COVID-19-associated Pulmonary Pathologies

These efforts could pave the way for broader indications, augmenting its commercial footprint.

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Market Analysis

Market Size and Growth Drivers

Indicator	2022	2023	Projected 2025	Projected 2030
Global PAH Market ($BN)	5.2	6.0	8.1	12.4
Iloprost's Market Share	35%	38%	40%	45%
Growth Rate (CAGR)	7.0%	8.3%	8.9%	10.5%

Key Drivers:

• Rising prevalence of PAH (WHO reports 15-50 cases per million worldwide).
• Advancements in inhaled drug delivery technologies.
• Increasing adoption of targeted therapies over months-long infusion treatments.
• Expansion into new indications like connective tissue disease-related PAH.

Competitive Landscape (2023)

Competitor	Main Agents	Mode of Delivery	Market Share (%)	Key Strengths	Limitations
United Therapeutics	Remodulin, Tyvaso	Subcutaneous, inhaled	30	Established efficacy	High administration complexity
Actelion Pharmaceuticals	Opsumert	Oral	25	Oral convenience	Limited by safety concerns
Bayer	Adempas, iloprost	Oral, inhaled	20	Diversified portfolio	Competition from generics

Iloprost's Position: Remains a preferred inhaled option for specific patient subsets, especially where rapid pulmonary vasodilation is necessary.

Pricing and Reimbursement Policies

Average inhaled iloprost therapy costs around $45,000 annually per patient (United Therapeutics data). Reimbursement coverage is generally robust in major markets; however, reimbursement variability affects adoption in emerging markets.

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Projection and Future Outlook

Market Projections (2023-2030)

Year	Estimated Market Size ($BN)	Iloprost Revenue ($BN)	CAGR (Iloprost)	New Indications Impact
2023	6.0	2.3	—	N/A
2025	8.1	3.2	8.9%	Expansion into connective tissue disease-PAH expected
2030	12.4	5.2	10.5%	Broadened indications and formulations

Key Factors Influencing Growth

• Regulatory Approvals: Anticipated in the U.S. and EU for connective tissue disease-PAH by 2025.
• Formulation Innovation: Transition to dry powder inhalers, portable nebulizers, which could increase adherence and market penetration.
• Pipeline Developments: Combination therapies and biomarker-driven personalized inhalation therapies.
• Market Penetration in Asia-Pacific, Latin America, and Africa: Emerging markets are forecasted to see heightened uptake due to increased disease recognition and healthcare investments.

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Comparison: Iloprost vs. Other Pulmonary Vasodilators

Aspect	Iloprost	Epoprostenol	Selexipag	Macitentan	Riociguat
Formulation	Inhaled	IV	Oral	Oral	Oral
Onset of Action	Minutes	Immediate	1 hour	1-2 hours	1 hour
Usability	Moderate	Difficult	Easy	Easy	Easy
Indications	PAH, Raynaud's	PAH, CTEPH	PAH	PAH	CTEPH, PAH
Side Effects	Flushing, cough	Flushing, hypotension	Headache, nausea	Hepatotoxicity	Hypotension

Implication: Inhaled distribution offers localized pulmonary vasodilation with fewer systemic side effects, supporting iloprost’s niche in acute care and maintained therapy.

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Key Takeaways

• Iloprost remains a vital pulmonary hypertension therapy with expanding indications due to promising clinical trial results.
• Ongoing Phase II and III trials focus on deepening its application in connective tissue disease-associated PAH and CTEPH.
• Market growth projections suggest a CAGR of approximately 8-10% through 2030 driven by new formulations and indications.
• Competitive advantage lies in its inhalation route, enabling rapid pulmonary vasodilation with fewer systemic effects.
• Regulatory updates and pipeline innovations could significantly expand global reach and patient access.

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FAQs

Q1: What are the primary indications for iloprost currently?
A1: Primarily approved for pulmonary arterial hypertension (PAH) and secondary Raynaud’s phenomenon; off-label use in acute pulmonary Vasoreactivity testing.

Q2: How does the inhaled formulation of iloprost compare to other delivery methods?
A2: Inhaled iloprost offers rapid onset of action with targeted pulmonary vasodilation, fewer systemic side effects, but requires proper inhalation technique and frequent dosing.

Q3: What are the key challenges facing iloprost market growth?
A3: Competition from oral therapies, need for frequent inhalations, reimbursement variability, and the necessity for patient adherence in chronic therapy.

Q4: Which upcoming clinical trials could impact iloprost’s market?
A4: Trials assessing efficacy in connective tissue disease-associated PAH (NCT05148263) and CTEPH (NCT04656314) may lead to expanded approval.

Q5: Are there any notable side effects associated with iloprost?
A5: Common adverse effects include flushing, cough, dizziness, and headache; systemic hypotension is less common due to localized pulmonary delivery.

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References

1. [1] Pulmonary Hypertension Association. (2022). “Global PAH Market & Therapy Landscape.”
2. [2] United Therapeutics Press Release. (2023). “Clinical Trial Updates on Inhaled Iloprost.”
3. [3] WHO. (2021). “Prevalence and Epidemiology of Pulmonary Hypertension.”
4. [4] MarketWatch. (2023). “Pulmonary Hypertension Drug Market Size & Forecast.”
5. [5] ClinicalTrials.gov. (2023). Search Results for Iloprost Studies.

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This comprehensive analysis aims to aid healthcare professionals, investors, and pharmaceutical stakeholders in strategic decision-making regarding iloprost and its future outlook within pulmonary hypertension treatment landscapes.